Clinical Trial News
Saruparib Shows Promise in HRR-Deficient Breast Cancer
- Saruparib, a selective PARP1 inhibitor, demonstrates a promising objective response rate in patients with homologous recombination repair (HRR)-deficient breast cancers.
- The Phase I/II PETRA trial evaluated saruparib in previously treated HRR-deficient breast, ovarian, pancreatic, or prostate cancer patients, showing manageable safety.
- Results indicated a 48.4% objective response rate and a median progression-free survival of 9.1 months among breast cancer patients treated with 60 mg saruparib.
- Saruparib's properties enable patients to reach high drug exposure levels, potentially improving efficacy due to fewer dose interruptions and reductions.
Highlighted Clinical Trials:
AstraZeneca
Posted 11/12/2020
AstraZeneca
Posted 11/21/2023
FDA Accepts Shorla Oncology's NDA for Oral Liquid Leukemia Drug SH-201
- The FDA has accepted Shorla Oncology's New Drug Application (NDA) for SH-201, an oral liquid formulation of a chemotherapy drug used to treat certain forms of leukemia.
- The FDA's decision on the approval of SH-201 is expected by November 30, 2024, under the Prescription Drug User Fee Act (PDUFA).
- SH-201 offers an alternative treatment option for leukemia patients who have difficulty swallowing tablets or other solid dosage forms, addressing an unmet need.
- Shorla Oncology acquired the rights to SH-201 in July 2023 and is preparing for the launch, commercialization, and sales of the drug in the U.S.
Single Injection of Zilebesiran Reduces Blood Pressure by Up to 12 mmHg in Phase 2 Trial
- A single subcutaneous injection of investigational drug zilebesiran reduced systolic blood pressure by 4-12 mmHg at three months when added to standard antihypertensive medications in patients with uncontrolled hypertension.
- The KARDIA-2 study enrolled 672 patients and demonstrated statistically significant blood pressure reductions that persisted at six months despite addition of other antihypertensive treatments.
- Zilebesiran works by reducing angiotensinogen production in the liver and requires dosing only every three to six months, potentially improving medication adherence compared to daily oral treatments.
- No new serious safety concerns were observed regarding potassium elevations, kidney injury, or low blood pressure when zilebesiran was combined with standard antihypertensive medications.
Investigational mRNA Vaccine Shows Promise in Fighting Pancreatic Cancer
A phase 1 clinical trial of an mRNA-based cancer vaccine, autogene cevumeran, has shown potential in stimulating a lasting immune response against pancreatic cancer, with some patients showing no recurrence up to three years post-treatment. The vaccine, developed through a collaboration between BioNTech and Genentech, targets neoantigens specific to each patient's tumor, aiming to train the immune system to recognize and attack cancer cells. A phase 2 trial is underway to further evaluate the vaccine's efficacy and safety.
Highlighted Clinical Trials:
Genentech, Inc.
Posted 10/18/2023
FDA Accepts PTC Therapeutics' NDA for Sepiapterin to Treat PKU
PTC Therapeutics, Inc. has announced that the FDA has accepted for filing the New Drug Application (NDA) of sepiapterin, aimed at treating pediatric and adult patients with phenylketonuria (PKU). This marks a significant step towards providing a new treatment option for PKU patients, supported by promising clinical trial data.
Verastem Oncology's Avutometinib and Defactinib Combination Receives FDA Priority Review for Recurrent KRAS Mutant LGSOC
- The FDA has accepted Verastem Oncology's NDA for avutometinib plus defactinib with Priority Review for recurrent KRAS mutant low-grade serous ovarian cancer (LGSOC).
- The PDUFA target action date is set for June 30, 2025, potentially bringing the first FDA-approved treatment for this specific LGSOC subtype.
- The NDA is supported by Phase 2 RAMP 201 trial data, showing substantial overall response rates and tolerability in patients with recurrent KRAS mutant LGSOC.
- Verastem Oncology also exercised its option to license VS-7375, a KRAS G12D inhibitor, with plans to initiate Phase 1/2a study in mid-2025.
Highlighted Clinical Trials:
Verastem, Inc.
Posted 3/18/2024
Verastem, Inc.
Posted 4/12/2022
Verastem, Inc.
Posted 3/22/2023
Verastem, Inc.
Posted 12/21/2020
Odronextamab Demonstrates High Efficacy in Lymphoma Treatments at ASH Meeting
New data presented at the 66th ASH Annual Meeting highlights odronextamab's potential in treating various B-cell non-Hodgkin lymphoma subtypes, showing complete responses in untreated follicular lymphoma and durable responses in diffuse large B-cell lymphoma post CAR-T therapy.
CARVYKTI® Approved as First BCMA-Targeted Therapy for Relapsed/Refractory Multiple Myeloma After One Prior Line
- CARVYKTI® (ciltacabtagene autoleucel) gains FDA approval for relapsed or refractory multiple myeloma after just one prior line of therapy.
- The approval is based on the Phase 3 CARTITUDE-4 study, which showed a 59% reduction in disease progression or death compared to standard therapies.
- This approval expands access to CARVYKTI®, offering a potential treatment-free period for more patients with multiple myeloma early in their treatment journey.
- CARVYKTI® is a personalized, one-time infusion that harnesses a patient's own T-cells to fight the disease, targeting B-cell maturation antigen (BCMA).
Highlighted Clinical Trials:
Janssen Research & Development, LLC
Posted 6/12/2020
FDA Approves Expanded Use of TRIKAFTA for Cystic Fibrosis Treatment
The U.S. FDA has approved the expanded use of TRIKAFTA for treating cystic fibrosis in patients aged 2 and older with specific CFTR gene mutations, making approximately 300 more people eligible for this treatment. The approval includes updates to safety information regarding liver injury and liver failure.
Meta-Analysis Confirms Superior Efficacy of Perioperative Immunotherapy Plus Chemotherapy in Resectable NSCLC
- A comprehensive meta-analysis of five phase III trials involving 2,855 patients demonstrates that perioperative immunotherapy combined with chemotherapy significantly improves overall survival, event-free survival, and pathological complete response rates compared to chemotherapy alone in resectable non-small cell lung cancer.
- The combination therapy reduced the risk of death by 32% (HR=0.68) and achieved a 7.54-fold higher pathological complete response rate, with particular benefits observed in current and former smokers and patients with EGFR-mutation negative tumors.
- While the treatment increased grade 3+ adverse events and immune-related complications, it maintained surgical feasibility with 100% R0 resection rates and no increase in treatment-related deaths.
Highlighted Clinical Trials:
BeiGene
Posted 5/29/2020